NCT02025491.

Background The treatment of cutaneous leishmaniasis (CL) in Brazil by pentavalent antimony (Sb v) is associated with a high rate of failure. Oral miltefosine in monotherapy has proven high efficacy in CL caused by L. braziliensis with a cure rate of 75%. A combined treatment with GM-CSF and miltefosine was tested to increase the cure rate and decrease the healing time Methods This is a randomized and double-blind clinical trial to evaluate the efficacy of miltefosine combined with topical GM-CSF (M+GM) versus miltefosine and placebo (M+P) versus standard Sb v in the treatment of 133 patients with CL caused by L. braziliensis in Bahia, Brazil Results The final cure rate at 180 days after the initiation of treatment was 44.4% in the Sb v group, 76.6% in the M+P group (P= 0.003 versus Sb v), and 75.6% in the M+GM group (P= 0.004 versus Sb v). By survival curve analysis median healing time for cure was 102 for Sb v group and 60 days for both miltefosine groups (P= 0.0009). During the 6 months follow-up period, four relapses were documented, one in the Sb v group (2%), one in the M+P group (2%) and two in the M+GM group (5%). Adverse events were documented in 65% of subjects from Sb v group, 79% of M+GM group and 76% of M+P group Conclusions Miltefosine is more effective than standard Sb v for the treatment of CL caused by L. braziliensis in Brazil and accelerate the healing time of CL. The association of Miltefosine with GM-CSF do not improve therapeutic outcome